Successful Natalizumab Treatment of Two Female Individuals With Susac Syndrome.
European journal of neurology March 1, 2025 Agni M Konitsioti, Rafael Grajewski, Mark Schlamann et al. 1 citation
Susac syndrome is a rare autoimmune disorder affecting the brain, retina, and inner ear, often causing encephalopathy, vision loss, and hearing problems. Two patients with Susac syndrome received off-label treatment with natalizumab, a drug typically used for multiple sclerosis. Over up to 22 months of follow-up, both patients showed clinical stabilization and reduced disease activity on MRI and retinal angiography. The treatment was well tolerated without significant side effects. Natalizumab may be a potential off-label therapy for Susac syndrome, but more research is needed to confirm its safety and effectiveness.